Clinical Insights
A Family’s Journey with Prader-Willi Syndrome
Maria Picone, co-founder and CEO of the TREND Community, and mother of a 14-year-old with Prader-Willi syndrome (PWS), discusses her family’s journey with PWS, including why more research and better data are so important. Understanding Prader-Willi...
3D Printed Treatment Options | The CheckRare Brief
In this episode, we discuss a new formulation of treatment for HAE, the FDA approval of Voceryq for recurrent respiratory papillomatosis, and 3D printed treatment options.
Molecular Changes Associated With Teprotumumab Treatment in Thyroid Eye Disease
Sarah Cheng, MD, PhD, a clinical oculoplastic fellow at the University of California, San Diego, discusses findings from an exploratory study examining the biological changes in orbital tissue associated with Tepezza (teprotumumab-trbw) treatment. The findings were presented at the 2026 American Academy of Ophthalmology (AAO) Annual Meeting in New Orleans, Louisiana.
FDA Grants Accelerated Approval to Voceryq for Recurrent Respiratory Papillomatosis
The US Food and Drug Administration (FDA) has granted accelerated approval to Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd), previously referred to as INO-3107, to reduce the frequency of surgery in adults with recurrent respiratory papillomatosis (RRP).
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Oxbryta (Voxelotor) Clinical Study Analysis Strives to Get Sickle Cell Disease Treatment Back on Market
A recent clinical study analysis of Oxbryta (voxelotor), globally withdrawn in 2024, was submitted to the FDA with the intent of getting voxelotor back on the market. Sickle cell disease (SCD) is a...
Efficacy of Efgartigimod To Treat Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): ADHERE Trial
Hans Katzberg, MD, Professor of Neurology at the University of Toronto and Neurologist at Toronto General Hospital, discusses post hoc analyses from the ADHERE clinical trial in chronic inflammatory...
Key Features of PLGD-1
Angela Zhu, MD, is an Ophthalmologist and Clinical Assistant Professor, Pediatric & Adult Cornea/Cataract/External Diseases, at Bascom Palmer Eye Institute in Miami. In this interview with...
Primary Analysis of KYV-101 for the Treatment of Stiff Person Syndrome
Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses primary analysis of KYV-101 for the treatment of stiff person syndrome (SPS). SPS is a...
Narcolepsy Breakthroughs and Emerging Therapies
Emmanuel Mignot, MD, PhD, is one of the pioneers in the study of narcolepsy and served a vital role in discerning the genetic cause of this rare and underdiagnosed disorder. Dr. Mignot, Professor of...
Accelerated Approval Granted to Beqalzi (Sonrotoclax) for Mantle Cell Lymphoma
The US Food and Drug Administration (FDA) has granted accelerated approval to Beqalzi (sonrotoclax) for the treatment of adults with relapsed or refractory (R/R) mantle cell lymphoma (MCL) after at...
Two-Year Results of Crenessity (Crinecerfont) in the Treatment of Congenital Adrenal Hyperplasia
Richard J. Auchus, MD, PhD, Professor of Internal Medicine and Pharmacology at the University of Michigan Medical School, discusses two-year results of Crenessity (crinecerfont) in the treatment of...
Ecopipam as a Treatment for Tourette Syndrome: Phase 3 Clinical Trial Results
Kinga Tomczak, MD, PhD, Program Director of the Tic Disorders and Tourette Syndrome Program at Boston Children’s Hospital and Assistant Professor of Neurology at Harvard Medical School, discusses...
Bizengri (Zenocutuzumab) Approved for NRG1 Fusion-Positive Cholangiocarcinoma Under the FDA’s National Priority Voucher Program
The US Food and Drug Administration (FDA) has approved Bizengri (zenocutuzumab) for the treatment of adults with NRG1 fusion-positive cholangiocarcinoma. Cholangiocarcinoma is a rare, aggressive...
FDA Expands Indication of Vyvgart (Efgartigimod Alfa) for Adults With Seronegative Myasthenia Gravis
The US Food and Drug Administration (FDA) has approved Vyvgart (efgartigimod alfa) and Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase) for the treatment of adult patients who have anti-MuSK-Ab...
New Follow-Up Data of KYV-101 Treatment for Patients With Myasthenia Gravis
Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses new follow-up data on KYV-101 treatment for patients with myasthenia gravis (MG). ...
Diagnosis and Management of Plasminogen Deficiency
Amol Sura, MD, Foster Center for Ocular Immunology, Dept of Ophthalmology, Duke University, Durham, North Carolina, discusses the diagnosis and management of plasminogen deficiency, a rare condition...
8-Week Follow-Up Data of RAP-219 for the Treatment of Focal Onset Seizures
William W. Motley, MD, RAP-219 Program Leader at Rapport Therapeutics, discusses follow-up data of RAP-219 for the treatment of focal onset seizures. Focal onset seizures refer to...
GEMZ Clinical Trial of Fenfluramine in Patients With CDKL5 Deficiency Disorder
Brian Moseley, MD, Senior Medical Director at UCB, discusses the GEMZ clinical trial of fenfluramine in patients with CDKL5 deficiency disorder (CDD). CDD is a rare genetic disorder...
CareCompass: Online Tool for Caregivers of Dravet and Lennox-Gastaut Syndrome
Colin Lake, Head of Digital Business Transformation Neurology at UCB, discusses CareCompass, an online tool for caregivers of patients with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS)....
FDA Approves Extended Release Ruxolitinib for Three Rare Hematologic Disorders
The US Food and Drug Administration (FDA) has approved Jakafi XR (ruxolitinib) extended-release tablets for the treatment of three rare hematologic conditions. The indications include the treatment...
CureFest 2026
Dena Sherwood, mother to a neuroblastoma survivor and Founder of Arms Wide Open Childhood Cancer Foundation, discusses CureFest 2026. Ms. Sherwood and her family founded the Arms Wide...
Positive Results with Cemdisiran, an RNAi Mediated Therapy, in Patients With Myasthenia Gravis
Tuan Vu, MD, Professor, Department of Neurology at the University of South Florida, discusses positive results of cemdisiran in patients with generalized myasthenia gravis (gMG). MG is...
Effects of Rodatrisat Ethyl on Activity in Patients With Pulmonary Arterial Hypertension
A study published in the Annals of the American Thoracic Society analyzed results from the ELEVATE-1 clinical trial testing the effects of rodatristat ethyl on daily activity in patients with...
An Overview of Systemic Mastocytosis
Dareen D. Siri, MD, FAAAAI, FACAAI is a board-certified allergist and immunologist practicing at Midwest Allergy Sinus Asthma, based in central Illinois. She talked with CheckRare about a unique,...
Systemic Mastocytosis: Recognition, Diagnosis, and Clinical Management
Highlighting the early signs of Systemic Mastocytosis (SM), criteria and tools, and the importance of timely referral/testing
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TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
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Highlights from The Liver Meeting 2026 | The CheckRare Brief
Results of the Phase 3 OASIS Study of Oral Olorofim
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What should you know about myasthenia gravis?
From understanding the underlying disease mechanisms to exploring FDA-approved treatment options and ongoing clinical research, staying informed is essential for the rare disease community.
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UCB Study Highlights the Burden of Ocular Myasthenia Gravis
Living With Ocular Symptoms of Myasthenia Gravis
Steven Nathan, MD, MBBCh, FCCP, Director of the Advanced Lung Disease and Lung Transplant Program at Inova Fairfax Hospital, discusses findings from a post-hoc analysis of the phase 3 TETON-2 study, presented at the CHEST 2026 Annual Meeting.
View the full interview at Read morehttps://checkrare.com/inhaled-treprostinil-reduces-multiple-disease-progression-events-in-ipf/
What's the latest research in rare liver conditions?
Get updates from The Liver Meeting 2026 and more on this week's episode of The CheckRare Brief at https://checkrare.com/highlights-from-the-liver-meeting-2026-the-checkrare-brief/
What's the latest research in rare liver conditions?
Get updates from The Liver Meeting 2026 and more on this week's episode of The CheckRare Brief at https://checkrare.com/highlights-from-the-liver-meeting-2026-the-checkrare-brief/
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026 | The CheckRare Brief
Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven (UZ Leuven) in Belgium and Principal Investigator of the OASIS study, discusses positive phase 3 trial results evaluating oral olorofim in patients with invasive aspergillosis.
View the full interview at Read morehttps://checkrare.com/phase-3-oasis-study-shows-promise-of-oral-olorofim-for-invasive-aspergillosis/
Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven (UZ Leuven) in Belgium and Principal Investigator of the OASIS study, discusses positive phase 3 trial results evaluating oral olorofim in patients with invasive aspergillosis.
View the full
Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
Results of the Phase 3 OASIS Study of Oral Olorofim
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
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